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Michael J. Fox Foundation Commits up to $3.8 Million to Develop Gene Silencing Neuroprotective Treatment for Parkinson's Disease
Date:1/4/2008

NEW YORK, Jan. 4 /PRNewswire-USNewswire/ -- The Michael J. Fox Foundation has committed up to $3.8 million for the development of a gene silencing therapeutic to treat Parkinson's disease by reducing expression of the protein alpha-synuclein. A team of researchers led by Matt Farrer, PhD, of Mayo Clinic Jacksonville (Florida) with collaborators at Alnylam Pharmaceuticals and The Parkinson's Institute and Clinical Center will work to optimize a small interfering RNA (siRNA)-based therapeutic that could slow or stop the progression of Parkinson's disease. If successful, the project could result in an entirely new class of drug targeting the alpha-synuclein gene, which has proved difficult to modulate using traditional small-molecule therapeutics.

The work is being funded under the Foundation's LEAPS (Linked Efforts to Accelerate Parkinson's Solutions) 2007 initiative. LEAPS 2007 was funded with a lead gift from the Edmond J. Safra Philanthropic Foundation. The Edmond J. Safra Philanthropic Foundation has been one of the most steadfast supporters of The Michael J. Fox Foundation since its inception.

"Available Parkinson's treatments mask symptoms but do nothing to halt or slow underlying disease progression," said Katie Hood, chief executive officer of MJFF. "More and more scientific evidence supports the hypothesis that lowering alpha-synuclein levels in the brain could achieve the so-called 'Holy Grail' of PD research, a neuroprotective therapy. But no drugs have been identified to date that are capable of reducing alpha-synuclein expression; new approaches are needed. This LEAPS grant is characteristic of how The Michael J. Fox Foundation goes about its work -- making big bets on fresh ideas with potential to impact patients' quality of life."

While its normal function in the brain remains unknown, the accumulation of excess alpha-synuclein has been shown to be the cause of some familial forms of PD. Clinical, genetic and experiment
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SOURCE Michael J. Fox Foundation
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